In a disappointing turn of events for patients with amyotrophic lateral sclerosis (ALS), the maker of the drug Relyvrio has announced its decision to remove the medicine from the market. Amylyx Pharmaceuticals, the company behind the drug, acknowledged that it did not provide any benefits to ALS patients, prompting them to halt sales and marketing in the United States and Canada. This move comes as a blow to the ALS community, which had hoped for more effective treatments to combat the deadly neurological condition.
Relyvrio received approval from the Food and Drug Administration (FDA) in September 2022 after a long campaign by ALS patients advocating for its use. However, a recent large-scale study revealed that the drug failed to show any improvements in survival or other health measures such as muscle strength or walking ability. With this disappointing outcome, Amylyx Pharmaceuticals made the difficult decision to withdraw the drug from the market.
The withdrawal of Relyvrio leaves only three ALS medicines available to patients in the United States, with only one of them shown to extend survival by several months. This limited range of treatment options underscores the urgent need for more effective therapies for ALS. Patients and advocates have long urged federal agencies to fund and approve experimental therapies, and the failure of Relyvrio is a setback for their efforts.
As part of its restructuring efforts, Amylyx Pharmaceuticals also announced that it will lay off 70% of its workforce, affecting more than 350 employees. Despite this setback, company executives plan to continue studying Relyvrio and another experimental drug for several rare diseases, including Wolfram syndrome.
The FDA’s approval of Relyvrio in 2022 was controversial, as it was primarily based on results from a small mid-stage study that faced criticism from some of the agency’s own internal scientists. Typically, the FDA requires two large late-stage studies demonstrating clear benefits before granting approval. However, in the case of Relyvrio, the agency cited the serious and life-threatening nature of ALS as a reason for regulatory flexibility.
Relyvrio itself is a combination of two older drugs – one used for liver disorders and the other associated with traditional Chinese medicine. The drug had faced criticism for its high price tag, with a year’s supply costing $158,000. Disappointingly, sales did not meet expectations, with some patients discontinuing the medicine after only a few months.
The removal of Relyvrio from the market highlights the challenges in developing effective treatments for ALS. This devastating disease gradually destroys nerve cells and connections needed for basic functions like walking, talking, speaking, and breathing. Most patients with ALS die within three to five years of diagnosis, underscoring the urgent need for more effective therapies.
In conclusion, the withdrawal of Relyvrio from the market due to its lack of benefits for ALS patients is a significant setback for both patients and advocates. With limited treatment options available, the ALS community continues to face an uphill battle in their fight against this deadly disease. The failure of Relyvrio underscores the need for continued research and development of effective therapies for ALS, providing hope for the future of those affected by this devastating condition.
